NS Merck Serono Supplement April 2016

This supplement to the New Statesman is sponsored by Merck Serono Limited (“Merck”).

The views expressed are those of the authors and do not necessarily represent the views of Merck.

with George Freeman, Hilary Newiss, Andrew Dillon,

Norman Lamb, Jo Churchill, Lord Hunt, Elisabeth Prchla and Richard Murray

In association with

01 Merck cover.indd 1 29/03/2016 11:09:17

2 | NEW STATESMAN | 1-7 APRIL 2016

HEADINGFACTS AND FIGURESSH

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Health check

Sources: Association of the British Pharmaceutical Industry, 2015; University of York; National Institute for Health and Care Excellence, 2015

http://www.abpi.org.uk/industry-info/knowledge-hub/medicines/Pages/nice.aspx (accessed March 2016)

https://www.nice.org.uk/news/blog/carrying-nice-over-the-threshold (accessed March 2016)


Amount pharmaceutical industry spent in 2015 to help pay for medicines for NHS patients

(PPRS rebate)

£619m

The National Institute for Health and Care Excellence (NICE) uses a threshold to determine cost-

effectiveness of new treatments. Less than £20k per year to extend quality of life is acceptable. Over £30k is not (an exception is made for end-of-life

criteria up to a £50k threshold)

< £20,000= fund

> £30,000 = reject

ICERIncremental

cost-effectiveness ratio

Cost of treatment

A(£)Cost of

treatment

B(£)Outcome oftreatment

A(QALY)

Outcome oftreatment

B(QALY)

Rate of recommendation

An example of how to calculate value

technologies are recommended by NICE

8/10

6/10cancer drugs are

recommended by NICE

Quality of Life

1.0

0.7

0.5

1 year 2 yearsT

ICER =

0.5 QALYs1.4 QALYs

B£9,600

A£15,000

£15,000-£9,6001.4 QALYs-0.5 QALYs

= 6,000 £QALYs

02 facts & figures V2.indd 2 29/03/2016 11:09:58

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CONTENTS

Access all areasThe means may be disputed but the ends are not. Policymakers, health professionals, patients and pharmaceutical companies all agree that the timely discovery and adoption of medicines that prolong and improve lives is an essential mission of health care.

Nowhere is this goal more fundamental than in the pursuit of cancer treatments. It is a motherhood issue. For evidence, look no further than two recent efforts to address it – the Cancer Drugs Fund, introduced by the coalition government in 2011 and about to be reconstituted; and the Accelerated Access Review,

commissioned by the Minister for Life Sciences, George Freeman, which, in his own words (see page 12), was launched to “ask a very simple but profound question: how do we adapt the UK landscape to this new world of 21st-century precision medicine?”

Yet the consensus only goes so far. The record of the Cancer Drugs Fund has been mixed (page six) and even those inside the last government are critical. Norman Lamb, a health minister until last May, writes on page 11 that “though well-intentioned, the Cancer Drugs Fund in its current form falls some way short

2 Facts and FiguresHealth checkSpend and value for money – how it all breaks down

4 View from Merck SeronoMaintaining life sciences in the UKSupport is still needed for a sector that promotes health and growth, writes Elisabeth Prchla

6 OverviewSlow road to the fast trackJon Bernstein looks at attempts to speed up access, while key voices offer timely advice

8 Professional vox popHow to ensure access to cancer medicinesHilary Newiss, Andrew Dillon, Jo Churchill and Richard Murray offer expert views on what the government should do

10 Jargon busterFrom AAR to QALYDecoded: the technical and industry terminology featured in this supplement

11 View from Westminster ITime to rethink our blunt access rulesThe Cancer Drugs Fund is causing the same distress it was set up to avoid, notes Norman Lamb

12 Interview“A quiet revolution in precision medicine”Life Sciences Minister George Freeman on the CDF, AAR and the future of drug adoption

15 View from Westminster IITime to end slow NHS adoptionThere is an alarming disparity of access in the UK compared to other countries, writes Lord Hunt

Value for money is vital in health care George Freeman gazes into the future

of . . . a scheme that is equitable, evidence-based and sustainable”.

Other voices weighing in on the subject include Richard Murray from the King’s Fund; Hilary Newiss, chair of National Voices; and the NICE chief executive, Andrew Dillon (pages eight and nine). The last word goes to Jo Churchill MP, a cancer survivor twice over. “In 2010, in my first speech in parliament as a cancer campaigner, I called for more to be done. We lagged behind many other countries in outcomes and unfortunately, although UK cancer survival is at its highest, we still do.” l

How to clear the barriers to access

The paper in this magazine originates from timber that is sourced from sustainable forests, responsibly managed to strict environmental, social and economic standards. The manufacturing mills have both FSC and PEFC certification and also ISO9001 and ISO14001 accreditation.

This supplement to the New Statesman of which this article forms part is sponsored by Merck Serono Limited (“Merck”). The views expressed in this article are those of the author and do not necessarily represent the views of Merck.

First published as a supplement to the New Statesman of 1-7 April 2016. © New Statesman Ltd. All rights reserved. Registered as a newspaper in the UK and US.

This supplement and other policy reports can be downloaded from the NS website at newstatesman.com/page/supplements

The Nationaand Care Exca threshold

effectiveness othan £20k per of life is accept(an exception

criteria up t

< £=

> £3

ICERIncremental

cost-effectiveness ratio

A(£) B(£)Outcome oftreatment

A(QALY)

Outcome oftreatment

B(QALY)

Rate of recommendation

An example of how to calculate value

technologies are recommended by NICE

8/10

Quality of Life

1.0

0.7

0.5

1 year 2 yearsT

ICER =

0.5 QALYs1.4 QALYs

B£9,600

A£15,000

£15,000-£9,6001.4 QALYs-0.5 QALYs

= 6,000 £QALYs

2 126

03 contents+intro.indd 3 29/03/2016 11:12:26

“Life sciences is a jewel in the crown of our economy.” 1

David Cameron, Prime Minister

The life sciences sector is a major contributor to the UK economy. The sector (including pharmaceuti-

cals, biotechnology, medical technology and diagnostics) generates an estimat-ed annual turnover of £56bn and em-ploys over 183,000 people in more than 4,398 companies2.

Merck is a pioneering company with a unique combination of businesses across health care, life science and performance materials. In 2014, our global research and development investment totalled €1.7bn3. We focus on combining specialist and high-quality products to create solutions in health care that advance technologies for life, including the development of personalised medicines for the treatment of cancers and developing biomarkers that match effective treatment to individual patients. Most recently, our research in immuno- oncology is challenging the di-rection of cancer care as we know it by exploring how the immune system rec-ognises cancer cells and destroys them.

The life sciences contribution to the UK economy has been recognised by suc-cessive governments, which have intro-duced policies to help support sustainable growth in the sector and to maintain the UK’s position as an attractive destination for research and development investment.

However, there remains a major con-cern in the UK as to how some medicines are evaluated and assessed through tech-nology appraisals which could ultimately affect the biopharmaceutical industry’s willingness to invest in the sector. We should ask on both counts if the UK can learn anything from initiatives and sup-port mechanisms that are in place in other countries.

Meanwhile the Accelerated Access Re-view (AAR) has been welcomed by the pharmaceutical industry as an opportuni-ty to speed up access to innovative drugs, devices and diagnostics for NHS patients in the UK4. The AAR is intending to ex-plore ways to engage in earlier dialogue with industry, increase incentives to in-vest in UK health care and review reim-bursement and health system barriers to access and uptake. The much-anticipated final report is due this month.

The government must continue to support a sector that plays a crucial role in promoting the nation’s health and economic growth, writes Elisabeth Prchla

Maintaining life sciences in the UK

With the original Cancer Drugs Fund (CDF) having ended recently, the govern-ment is now presented with a challenge: the need to manage the growing number of innovative cancer medicines within a fixed budget, whilst acknowledging the importance of accelerating access under the AAR.

While initiatives such as the AAR make sense and are clearly very important, we would like to see further system-wide change and implementation in order to support access to innovative medicines.

However, the need for reform is not a new dilemma. The CDF was introduced in England in 2010 as a means to provide patients in the NHS with cancer drugs not approved by the National Institute for Health and Care Excellence (NICE) until a new model of value-based pricing (VBP) was introduced.5

Later in 2010, the government an-nounced its plans for the VBP mecha-nism, in recognition that NICE meth-odology for appraisals may not capture the full value of medicines in conditions such as cancer. This gave rise to a review of methods and the introduction of value-based assessment (VBA).

VIEW FROM MERCK SERONO


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In 2014, NICE concluded6 that further work needed to be undertaken to en-able changes to its appraisal methods and made proposals including:l An “agreement between NICE, NHS England and the Department of Health, on the NHS’s willingness to pay for new treatments, which would take account of any special cases, such as ultra-orphan conditions and cancer”.l “More productive sharing of risk be-tween companies and the NHS. The aim would be to progressively reflect the value of new treatments as our know-ledge of what they can offer to patients increases.”

Now, in 2016, the CDF (in its old form) is ending and is to be replaced by a system in which all new cancer drugs are to be referred to NICE for appraisal7. The origi-nal CDF list will be available while NICE makes an evaluation but will remain closed to new drugs pending the start of the new scheme in July this year.

The new CDF aims at providing pa-tients with access to promising new medicines (while the evidence is still emerging) through an annual “managed access” fund of £340m. Though the idea

is encouraging on many levels, how this fund will work in practice has been flagged as a concern by industry in its feedback to NHS England.8

Merck is committed to working closely with NHS England and appropriate bod-ies through ongoing appraisals within this evolving system. However, we share the view of the Association of the Brit-ish Pharmaceutical Industry 9(ABPI) that the finalised proposals confirm a seem-ingly reduced level of ambition from NHS England for providing NHS patients with access to the latest cancer medicines be-cause the NICE decision-making process remains largely unchanged.

In summary, life sciences contribute significantly to the UK economy; how-ever, access to innovative medicines is not always straightforward. A number of ini-tiatives are ongoing to address this issue but more needs to be done.

Merck will continue to work closely with NHS England and appropriate bod-ies to ensure that innovative treatments can be accessed by the right patients at the right time. lElisabeth Prchla is general manager (UK and ROI) at Merck

References:

1 Prime Minister’s speech on life sciences and opening up the NHS, 6 December 2011. Available at: https://www.gov.uk/government/speeches/ pm-speech-on-life-sciences-and-opening-up-the-nhs (accessed March 2016)

2 HM Government. Strength and Opportunity: Annual Update 2014. Available at: https://www.gov.uk/government/uploads/system/uploads/attachment_data/file/427769/BIS-15-224-BIS-strength-opp-2014.pdf (accessed March 2016)

3 Research and Development at Merck. http://ar2014.merckgroup.com/management-report/fundamental-information-about-the-group/ research-and-development-at-merck (accessed March 2016)

4 ABPI submission to Accelerated Access Review. September 2015. http://www.abpi.org.uk/our-work/value-access/Documents/ABPI%20Submission%20to%20the%20AAR.pdf (accessed March 2016)

5 Cancer Drugs Fund. Cancer Research UK. http://www.cancerresearchuk.org/about-cancer/ cancers-in-general/cancer-questions/cancer-drugs-fund (accessed January 2016)

6 NICE calls for a new approach to managing the entry of drugs into the NHS. 18 September 2014. http://www.nice.org.uk/news/press-and-media/ nice-calls-for-a-new-approach-to-managing-the-entry- of-drugs-into-the-nhs (accessed December 2015)

7 NHS England and NICE asks for views on the future direction of the CDF. 19 November 2015. https://www.england.nhs.uk/2015/11/19/cdf-consultations/ (accessed December 2015

8 Cancer Drugs Fund Consultation. NHS England. https://www.engage.england.nhs.uk/consultation/cdf-consultation/ (accessed March 2016)

9 ABPI comment on CDF announcement. ABPI 17 March 2016. http://www.abpi.org.uk/media-centre/newsreleases/ 2016/Pages/170316.aspx (accessed March 2016)

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OVERVIEW

To understand the imperatives, complexities and frustrations that accompany attempts to en-sure timely access to medicines in England, it’s worth retracing the

story, so far, of the Cancer Drugs Fund.Its roots can be traced back to 13 April

2010. That was the day David Cameron – then leader of the opposition – launched the Conservative Party’s manifesto in the build-up to the general election. On page 47 of the rather quixotically titled Invita-tion to Join the Government of Britain, and under the plaintive heading “Increase ac-cess to vital drugs and services”, came the following promise:

“NHS patients rightly expect to be among the first in the world to access ef-fective treatments, but under Labour they are among the last. We want more people to access the drugs and treatments that would prolong or improve their lives by reforming the way drug companies are paid for NHS medicines.

“Using money saved by the NHS through our pledge to stop Labour’s jobs tax, we will create a Cancer Drugs Fund to

enable patients to access the cancer drugs their doctors think will help them.”

In the event, Cameron’s party didn’t win an overall majority and was obliged to invite the Liberal Democrats to join the government of Britain. Despite this in-convenience, plans for the fund survived the coalition negotiations and in 2011 the CDF was introduced – the CDF being a £200m-a-year pot of money designed to bypass medicines regulator, the National Institute for Health and Care Excellence (NICE). If NICE rejected NHS adoption of certain cancer drugs – or had yet to ap-praise those drugs – the CDF could latter-ly grant access by underwriting the cost.

In the first two years of the fund’s existence, an estimated 44,000 patients benefited from it and by early 2016 it had helped more than 80,000 patients get access to medicines they might not have otherwise received.

Large-scale overspendingSo far, so encouraging. Yes, the initiative had some critics – the Scottish govern-ment ruled that introducing something

As government, industry and patients wrestle once again with the access issue, Jon Bernstein looks at recent attempts to address it, and

overleaf four leading voices on health offer some timely advice

Slow road to the fast track

similar north of the border was unneces-sary, while the NICE chairman, Sir Mike Rawlins, pointed out to the Commons health select committee in 2012 that there are “other rotten diseases apart from cancer. To limit it to cancer has always made me uncomfortable.”

Nevertheless, the Cancer Drugs Fund did appear to fulfil a need.

There remained a big problem, how-ever – overspending on a large scale. In 2015-16, the CDF was on course to spend £340m, or 70 per cent above the allocated budget. A public accounts com-mittee report in February criticised the management of the fund and suggested there was no evidence that it was benefit-ing patients.

And now the CDF – although it lives on in name – is about to be transformed dra-matically. Consultation for the new CDF began last November and by April it will become part of NICE’s assessment pro-cess for new drugs. It will no longer be a separate fund.

Mounting a robust defence for the new role of the CDF, Simon Stevens, chief ex-

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Drip, drip, drip: access to innovative pharmaceuticals, medical devices and in vitro diagnostics remains slow

ecutive of NHS England, said it would be used to “sort the wheat from the chaff” and would “fast-track exciting new drugs”. Launching the consultation, he argued: “Over the next five years we’re likely to see many new cancer drugs com-ing on to the worldwide market, some of which will be major therapeutic break-throughs and some of which will turn out to offer little extra patient benefit but at enormous cost.”

Meanwhile, Cancer Research UK’s di-rector of policy, Alison Cook, told the Times: “It’s been clear for some time that [the fund] is not sustainable. We’d like to see a single system for drugs approvals that can respond to patients’ needs.”

While Stevens’ and Cook’s arguments are perfectly reasonable, the CDF was set up with a specific purpose – to bypass the current processes – and few believe the old inefficiencies have been resolved. The Department of Health-initiated Ac-celerated Access Review – expected to report back its findings imminently – is testament to that. The goal of the AAR, originally announced last March, is “to

ensure that the UK is the fastest place in the world for the design, development and widespread adoption of medical in-novations. This will help stimulate new investment, jobs and economic growth to support a stronger NHS”.

Barriers to accessSo, why is access to treatment such a thorny issue? A July 2015 report by Moni-tor Deloitte, the Centre for the Advance-ment of Sustainable Medical Innovation

and the health think tank the King’s Fund attempts to answer that question.

Commissioned as part of the AAR pro-cess, the report identifies four “cross-cut-ting” challenges for access to innovative pharmaceuticals, medical devices and in vitro diagnostics.

The first challenge is what the report

authors describe as a lack of “alignment across stakeholders”. In other words, the goals of those involved in the process from development to adoption of new technologies and medicines don’t match up. Or, at best, there is misunderstanding between those involved. This happens between the national level and the local level where treatments are adopted.

There is also a lack of industry un-derstanding of NHS clinical priorities. Meanwhile, the voice of the patient is little heard.

The second challenge is the existence of counterproductive incentives. An ex-ample of this is where budgets are treated separately across NHS organisations and therefore potential costs become a barrier for adoption.

The third challenge is the failure to use data as evidence for the use of new treat-ments. This is sometimes because there is a lack of clarity around – or availability of – evidence required to make the case.

The final challenge, the report authors argue, is cultural. A lack of trust between the NHS and industry is, the report

The NHS overspent on drugs last year to the tune of £619m

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notes, “a key barrier to more collabora-tive working”. Other cultural barriers in-clude an aversion to risk, distrust of exter-nal data and evidence, and unconstructive competition between NHS organisations.

There are also barriers that are specific to the pharmaceutical industry. These, the report argues, are present at every stage from drug development to local commissioning and adoption. At the be-ginning of that journey – at the develop-ment stage – there is resistance to con-duct clinical research within the NHS or a lack of resources to do that research. The regulation stage is stymied by a lack of early dialogue, misaligned perceptions

of risk and benefit, and limited access to early approval. When it comes to national reimbursement, evidence requirements, different definitions of value and differing objectives among those involved all act as barriers to access. Local commissioning and adoption is stymied by process dupli-cation, budget silos, lack of accountability and lack of transparency about outcomes.

Measuring valueA theme that runs through all of these barriers and challenges is a lack of agree-ment over costs and values, either what constitutes value for money or, more fun-damentally, how to measure it.

The NHS does measure the cost effec-tiveness of the medicines it considers, or rather, the regulator NICE does. NICE uses a threshold to determine whether a new treatment represents value for mon-ey. It determines what it calls incremental cost-effectiveness ratios (ICERs) by calcu-lating the variance in the cost of two treat-ments and dividing that by the difference in outcomes as a result of those two treat-ments. Outcomes are based on what are called quality adjusted life years (QALYs), a trade-off between full health and time.

At present, if the ICER comes in at less than £20,000 per QALY, then it is deemed as favourable by NICE. In other words, access to the medicine is likely to be granted. If the calculation comes to more than £30,000 per QALY, then the medicine will be rejected. In exceptional circumstances – where life expectancy is short or the patient population is less than 7,000 – then the ICER threshold can be extended to £50,000 per QALY.

When it measures cost effectiveness, NICE is not only looking at the price of the

Why is access to treatment such

a thorny issue?

What should government do to ensure access to cancer medicines?Hilary Newiss “Bureaucracy can slow down research

projects, but it can’t be used to slow

down cancer growth.”

Person living with cancer

New drugs, devices and diagnostics

are developed to support the treatment

and care of patients, yet patients can

be conspicuously absent from the

development process.

If we want to ensure future access to

medicines, a good place to start is finding

out what is important to the people who

will be using those medicines. Cancer

patients across the country report that

poor communication is the aspect of

care most in need of improvement.

That means better information about

diagnosis and treatment options, and

better information about new medicines

and the decisions about their availability.

As a society, we are increasingly

empowered to take advantage of

innovation to manage our own care. We

can download apps, buy monitors, or

wear watches that help us to look after

our health. People are playing a more

active role in determining their own care.

This should extend to the development

of new medicines and treatments.

Decisions about access to new

medicines are based on whether that

treatment will bring about a positive

outcome. Who is better placed to

determine what a positive outcome

looks like than the people the treatment

is intended for?

Information is key. Patients need to

know when decisions will be made

about access to new treatments, what

new treatments have become available

for them, and how to challenge decisions

about access to new treatments. With

that information, patients can play an

important role in determining access.

Instead of leaving it to researchers,

regulators, commissioners, or politicians

to decide the pros and cons of a new

medicine, let’s involve the patients. lHilary Newiss is chair of National Voices, a coalition of 160 health and care charities

OVERVIEW

What should government do to ensure future access to cancer medicines?Richard MurrayEnsuring better access to cancer

medicines – and, indeed, innovative

products and technology – more widely

requires a number of interlocking

changes. First, it needs the NHS and

industry to work together even before

launch to identify, on one side, the key

needs of the NHS and, on the other, the

key upcoming innovations and then to

use this understanding to speed up the

development process. This information

can also be used to make the necessary

steps to prepare the NHS to make best

use of innovative products.

Second, development processes for

cancer drugs are already faster than

in the past. This creates a challenge

for the cost-effectiveness assessment

undertaken by NICE, as it means these

products almost inevitably have a less

complete evidence base on which it can

make a judgement. To overcome this

challenge, NICE needs greater flexibility

in handling this uncertainty in the

evidence base, for example, by making a

“conditional” judgement to be followed in

due course by a final judgement.

Third, we need greater flexibility in

pricing systems so the NHS can better tailor

payment to the product, rather than apply

the current one-size-fits-all approach. Many

other European countries already use a

wider array of approaches to pricing.

Fourth and finally, more help is

needed to support the NHS to make the

sometimes complex changes to care that

innovation can require. This all represents

a significant set of changes that will be

considered in the Accelerated Access

Review, due to report in the spring. lRichard Murray, director of policy, the King’s Fund


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medicines. This means, in theory at least, that a medicine that costs nothing may still be rejected. Why? A number of rea-sons. It may be that associated costs mean that the delivery outweighs the health benefits. Or that the use of the medicine (or other technology) may increase the use of other, costly health resources.

Money-back guaranteeGiven cost effectiveness is a critical fac-

tor in determining the use of medicines, the Department of Health and the phar-maceutical industry struck a deal known as the Pharmaceutical Price Regulation Scheme (PPRS).

Under the terms of this scheme, there is a cap of £8bn that the NHS will spend on branded medicines. Any expenditure above that amount will be reimbursed by the pharmaceutical companies. Accord-ing to figures from the Association of the British Pharmaceutical Industry, the NHS overspend last year amounted to £619m and in the next five years the industry ex-pects to pay back £4bn.

In Scotland, the approach is a little dif-ferent. Adaptive pricing reforms, intro-duced in 2014, allow pharmaceutical com-panies to drop their prices if approval has been rejected on the grounds of cost but not efficacy. This allows the drug compa-nies to be reconsidered without having to resubmit bids from scratch. “Often there is room for manoeuvre,” the Scottish Health Secretary, Alex Neil, has said.

Precision engineeringEmerging trends demand that a fresh ap-proach be sought in the way that new

What should government do to ensure access to cancer medicines?Andrew DillonIn this life sciences ecosystem, where

researchers, drug manufacturers,

regulators, evaluation agencies (such

as NICE), governments and health

systems need to engage successfully

for the interest of patients to gain

primacy, the struggle to align ambition

and optimise efficiency of process is a

continuing challenge. We are unlikely

ever to achieve full alignment or

optimal efficiency, but getting as close

as possible requires changes for all the

ecosystem’s inhabitants.

Health systems need to be clearer

about their ambition for access to new

therapies; regulators need to adapt their

evidence requirements and timelines

to be consistent with the nature of

the evidence for new products and

with risk appetite of their government

sponsor; agencies that assess value

have to sensitise their judgements to the

ambition and capacity of their health

system and the health system itself has

to be creative and flexible in finding

ways to manage the adoption of the new

products it wants into routine practice.

And companies? They have to change,

too. They have to understand better and

work with the ambitions of systems and

the constraints they are under. They

have to be more self-critical of the value

propositions they submit, they have to

price realistically and fairly, and they

must be prepared to match the creativity

and flexibility of their customers. lAndrew Dillon is chief executive of NICE

medicines are evaluated and introduced into the healthcare system in the UK. So believes Professor Sir John Bell, Regius Professor of Medicine at Oxford Univer-sity and chairman of the Accelerated Ac-cess Review’s external advisory group. Writing in the AAR’s interim report last autumn, he noted: “After 25 years of in-tense molecular characterisation of dis-ease, it is increasingly possible to define precisely the mechanisms responsible for mediating disease and consequently, how it can be best managed or treated.

“Our ability to categorise disease in patients much more precisely is likely to have a profound effect on clinical medi-cine as we identify sub-populations of pa-tients likely to obtain maximum benefit from therapies. Resources can thereby be concentrated on those who will ben-efit the most rather than the population at large. This focus on patient sub-pop-ulations, which is the basis of “precision medicine”, has already begun to affect the quality of new therapeutic products.”

In the same interim report, the AAR chairman, Sir Hugh Taylor, sounded a warning. Acknowledging the financial restraints facing the National Health

What should government do to ensure access to cancer medicines?

Jo ChurchillIn 2010, in my first speech in parliament

as a cancer campaigner, I called for more

to be done. We lagged behind many other

countries in outcomes and unfortunately,

although UK cancer survival is at its

highest, we still do.

Multiple issues, from late diagnosis and

the strain on diagnostics to radiotherapy

machines being beyond their sell-by date

are all a problem. But most challengingly

£1.2bn spent on the Cancer Drugs Fund

(CDF) in the past five years, with ever-

increasing demand, doesn’t guarantee

patient access to the best treatments.

We need a sustainable model.

The current NICE system is broken, our

drugs pathway is clogged and getting

drugs licensed in a timely manner is

impossible. This is challenging on two

counts. First, we have the most vibrant

life science industry, with huge advances in

genomics and informatics within reach and

second because, having developed the drugs

and trialled them here, other countries

seem to get speedier access to them. I hope

the Accelerated Access Review, which has

been looking at new approaches to access

to drugs and other countries’ models for

pricing and reimbursement, and the CDF

consultation, will start to bring forward

much-needed changes.

I want the future not to be a lottery. A

new CDF should be where we trial drugs,

collect evidence to ensure a drug or

treatment can be licensed and prescribed

or rejected speedily. That would be a truly

patient-centred approach. However, it is

patients and patient data that hold the key

to unlocking the future of cancer medicine.

Only by collating and building evidence of

cancer patients and their treatment can we

better understand the challenges and find

the solutions. lJo Churchill is the MP for Bury St Edmunds (Conservative) and a cancer survivor in 1995 and again in 2009


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Service, Taylor argued that England “will lose ground if research budgets are threatened, if our leading academic hos-pitals cannot afford to support research or use the latest drugs and technologies to pioneer developments in the treatment of the most complex conditions, or if the wider system is paralysed by the cost pressures it is facing and fails to invest in the change and innovation it requires to deliver better care to patients more effi-ciently and productively.”

Jargon buster

AAR – Accelerated Access Review

A review by the government to examine

how innovative medicines and medical

technologies can get to patients quicker.

Looks across the whole timeline of

medicines or technology, from research

and development to use.

ACD – Appraisal consultation

document

Preliminary guidance during a NICE

appraisal process from the appraisal

committee.

CDF – Cancer Drugs Fund

A dedicated monetary fund for oncology

drugs, to ensure patients get access to

cancer medicines that were deemed not

cost-effective by NICE (see below). A

Conservative Party manifesto promise

before the 2010 election, it was designed

to resolve the access issue temporarily.

Its introduction was prompted by the

UK’s poor outcomes and survival rates

compared to other European countries.

CRC – Colorectal cancer

Any cancer that affects the large bowel

(colon) or rectum. It is the fourth most

common cancer in the UK, affecting over

34,000 people a year.

DH – Department of Health

The government department that

provides strategic leadership for public

health, the NHS and social care.

FAD – Final appraisal decision

Final guidance from the appraisal

committee, which, if not appealed

against, will be written up in the final

NICE guidance.

HTA – Health care technology

assessment

A review of the clinical and economic

evidence of a treatment to show how

well a medicine works in relation to how

much it costs the NHS. A decision is

then taken about whether it offers value

for money. Technology appraisals take

one of two forms:

l A single technology appraisal (STA)

that covers a single technology for a

single indication.

l A multiple technology appraisal (MTA),

which normally covers more than one

technology, or one technology for more

than one indication.

ICER – Incremental cost-

effectiveness ratio

Used to measure the difference between

the current standard of care and a potential

new treatment. Arrived at by finding the cost

difference between the two and dividing it

by the difference in their effect.

NHS E – NHS England

Set up in 2013 as an arm’s-length body

(not part of the government) to provide

leadership on improving outcomes and

quality of care, and to oversee clinical

commissioning groups.

NICE – National Institute for

Health and Care Excellence

An arm’s-length body that assesses the

cost-effectiveness of medicines to the

NHS and provides guidance to the NHS

on whether they should be offered to

patients and how.

He added: “Patients can and should be at the centre of this stage. They and their representatives have been fully engaged in this review. For them, and in the best interests of the economy and our health system, we have to meet two challenges.

“First, we have to find a way of get-ting ahead of the curve in anticipation of the potentially transformative tech-nologies that are on the horizon, and in some cases already with us, so that these can be brought to our health system in

a sustainable way which benefits our patients, which taxpayers can afford, and which works for innovators themselves. Second, we have to energise our health system so that it is receptive to innova-tion and sees and uses new technologies as the best lever for delivering improved care with greater efficiency.”

Six years after the Cancer Drugs Fund made its debut appearance in the Con-servative Party manifesto, these challeng-es remain unresolved. l

OLS – Office of Life Sciences

A government department that provides a

link between the Department of Health and

the Department for Business, Innovation

and Skills to ensure that decisions on

health take into account the wider business

environment for life sciences companies

providing those products and services.

Personalised/stratified/

precision medicine

This is a medical model that separates

patients into different groups with medical

decisions, practices, interventions and/or

products being tailored to the individual

patient based on their predicted response

to treatment. The terms “personalised

medicine”, “precision medicine”, “stratified

medicine” and “P4 medicine” are all used

to describe this concept. In this model,

diagnostic testing is often employed for

selecting appropriate therapies based on

the context of a patient’s genetic content or

other molecular or cellular analysis.

PPRS – Pharmaceutical Price

Regulation Scheme

A deal between the pharmaceutical

industry and the Department of Health to

ensure that safe and effective medicines

are available to the NHS on reasonable

terms – and that a strong, profitable

pharmaceutical industry is maintained.

QALY – Quality Adjusted Life Year

The trade-off between full health and time.

A treatment that provides one year in full

health has a score of 1 QALY. A treatment

that provides one year with half normal

health or half a year in full health would

have a score of 0.5 QALY. These scores help

measure cost-effectiveness. l


tOVERVIEW


VIEW FROM WESTMINSTER I

When it was introduced in 2010, the Cancer Drugs Fund was welcomed as a beacon of hope for many people struggling to get ac-

cess to cancer medicines normally consid-ered too expensive for the NHS. Six years on, the fund has benefited tens of thou-sands of people. However, its ballooning budget and concerns over its management led to the scheme’s future being called into question. There is little doubt that it requires reform – but do the weaknesses reflect poor management alone, or prob-lems at a more fundamental level?

The wave of breakthrough cancer medicines emerging over the past five to ten years has been a triumph of mod-ern science. However, the UK has been notoriously slow in bringing these treat-ments to patients. In 2010, a report by the cancer tsar, Professor Sir Mike Richards, found that the UK ranked only 12th out of 14 developed countries for uptake of new cancer medicines.

I want the UK to be an international leader in access to cancer medicines. This can only be achieved through a scheme that is equitable, evidence-based and sustainable. Although well-intentioned, the Cancer Drugs Fund in its present form falls some way short of these principles – and the result has been a sudden axing of drugs from the fund’s list in recent months, causing the same distress and uncertainty that the initiative was set up to avoid.

Much of the criticism has focused on NHS England’s inability to get a grip on

its spiralling costs. As a recent report by the Commons public accounts committee points out, the fund’s budget has more than doubled from £175m in 2012-13 to £416m in 2014-15, with an overspend of £167m recorded over the past two years.

This cannot escape scrutiny at a time when NHS finances are under enormous pressure. Nor can it be justified by ap-pealing to improved survival and quality of life, following the dismal failure of the Department of Health and NHS England to gather adequate data on the scheme’s impact on patient outcomes. When budg-ets are under so much strain, the reality is that treatments funded by the NHS need to be shown to be both clinically benefi-cial and reasonable value to the taxpayer.

NHS England has proposed a reformed Cancer Drugs Fund, more closely aligned with the role of NICE, the body respon-sible for deciding which medicines are normally available in the National Health Service. Under the revised scheme, prom-ising new cancer drugs could be offered to patients while being evaluated for routine use in the NHS, with patient out-comes monitored for up to two years as part of NICE’s assessment.

I think this could provide part of the solution we need. Restoring the prin-ciple of decision-making based on evi-dence would place the fund on a more stable footing, and reduce the likelihood of costs proliferating in the future. In the long term, however, this should form part of a broader overhaul of the way in which innovative medicines are assessed for use in the NHS.

Despite best intentions, the Cancer Drugs Fund in its current form is causing the same distress and uncertainty that

it was set up to avoid, believes Norman Lamb

Time to rethink blunt access rules

There is a growing sense that a blunt health economic assessment based on data from clinical trials is an archaic meth-od of deciding whether patients should be granted or denied access to potentially life-saving or life-extending treatments. Allowing novel medicines to be tested more widely in the NHS would be a bold and highly welcome development, es-pecially if this is coupled with a stronger voice for clinicians and patients in NICE’s final decision-making process.

An assessment based more closely on patient outcomes would reduce uncer-tainty about the effectiveness of new drugs for NHS commissioners asked to pay for them from increasingly stretched budgets, while also properly rewarding pharmaceutical companies for valuable innovations. Most importantly, patients will benefit from tried-and-tested treat-ment options and improved health.

Ultimately, however, our ability to pay for innovative medicines will be increas-ingly restricted unless we are prepared to confront the severe funding crisis facing the NHS. That is why I have proposed the creation of an independent, cross-party commission to design a new, long-term settlement for the health and care system. Only by working together can we turn the UK into an indisputable world leader in access to medicines and cancer survival – today and for generations to come. lNorman Lamb is the MP for North Norfolk, and the Liberal Democrat spokesman on health. Between 2012 and 2015 he served as a health minister in the coalition government


11 Norman Lamb view from Westminster.indd 11 29/03/2016 11:14:38


INTERVIEW

George Freeman is the man tasked with helping the nation speed up the discovery and adoption of new treatments.

Here, he explains how he thinks it can be done

“A quiet revolution in precision medicines”

Earlier this year Merck Serono’s general manager for UK and Ire-land, Elisabeth Prchla, sat down for a wide-ranging conversation with the Life Sciences Minister,

George Freeman. The context of the dis-cussion were the imminent changes to the Cancer Drugs Fund and the release of the final Accelerated Access Review re-port commissioned last year by Freeman.

The theme running through the con-versation was the challenge of providing timely access to cancer medicines. This is an edited extract of Freeman’s views on these issues.

What do you believe are the

current key trends in the life

sciences and pharmaceuticals?

The pace of technological change in the life sciences sector, particularly the pace of genetic insights and the phenomenal computing power of informatics, is pro-foundly changing the way drugs are being

discovered and developed. It is in that context that the Prime Minister set out in 2011 in his groundbreaking speech on the strategy for the UK life sciences our ambi-tion to adapt the UK landscape to support this new model of 21st-century medicines research and development.

In the old model, which we have known for the past 50 years, medicines typically take ten to 15 years to be brought to market costing about $2bn, and go through a very long and complex pipeline of early-stage discovery, preclinical phase one, two and three trials, more up-marketing authori-sation, NICE assessment and National Health Service approval before a new medicine comes anywhere near a patient.

The transformations in genomics and informatics mean that we can com-pletely change the process and start with patient tissues, biopsies, data, genetics, and design and develop drugs around the patient – starting with the clinical assets and insights, rather than start-

ing with theoretical drug targets, tested through massive synthetic libraries and then a long chain process relying on ani-mals and computers.

What challenges do you believe

that this presents?

Whether you call it translational medi-cine, or precision or stratified medicine, there is a quiet revolution ongoing in drug research and development.

It is both hugely challenging for the ex-isting model, and sector, but also creates huge opportunities. It is challenging because this new generation of preci-sion medicines do not fit the one-size-fits-all blockbuster drug model which Big Pharma has developed and demands much more flexible models of assessment and pricing.

It is also challenging because, while the cost of drug discovery has not come down, the market sizes for targeted medicines get smaller and therefore the prices get

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higher. It is challenging for the NHS be-cause the more we discover about genet-ics, and the causes of diseases, the more specialist medicines come down the pipe-line, generating huge costs.

And what about the opportunities?

The opportunity is clear that here in the UK, with our world-class medical sci-ence, with a comprehensive NHS, and our leadership in genomics and informatics, we can be the global testbed for precision medicines. We are, however, in a race, a global race, and in January 2015, when I was invited by the White House health team to update them on the UK strategy, it was striking that President Obama an-nounced four days later, in his penulti-mate State of the Union Address, a mas-sive American commitment to the field of precision medicines.

This transformation also shifts, or changes, the key players in the landscape for research and development, so that

alongside traditional Big Pharma, we are seeing the emergence of specialist phar-ma and a new generation of biopharma companies. They are developing genom-ic biomarkers which allow a drug to be matched much more quickly to a particu-lar patient subgroup with almost guaran-teed efficacy.

This means that patient voice and con-sent for the use of data, tissues and genet-ics comes centre stage, which will mean a dramatically enhanced role of charities and patient groups in this new landscape. This is good news because these are the very same patient groups and charities currently campaigning for quicker access to innovative medicines, which is why I launched the independent Accelerated Access Review. Accelerated Access Review (AAR)

What do you consider to be a success

so far with the AAR? Do you believe

the AAR will solve the inherent issues

around accelerating access and uptake

that will enable continued industry

investment in the UK?

The AAR is a major review of the UK land-scape for developing, testing, adopting and reimbursing innovative medicines – as well as medical technologies, diagnos-tics and digital products. I launched it in order to ask a very simple but profound question: how do we need to adapt the UK landscape to this new world of 21st-century transformative medicine?

I want us to accelerate the speed with which we both discover and adopt new medicines and to take advantage of this new model. The Food and Drug Ad-ministration (FDA) critical path initia-tive, of about five to ten years ago, was a game-changer in the American research and development landscapes (40 per cent of new medicines are now approved through breakthrough designations in the United States).

The AAR is independent and I’ve t


asked it to address three key questions:l One, the funnel-in: how can we make it quicker and easier for innovators to get access to UK clinical assets for proof of concept?l Two, how can we update the mecha-nisms for the National Institute for Health and Care Excellence (NICE), Med-icines and Healthcare Products Regula-tory Agency (MHRA) and NHS England to appraise and, where appropriate, make available this whole new range of innova-tive drugs? l Three, the funnel-out: how can we in-centivise and remove barriers to quicker adoption of innovative drugs and diag-nostics devices, and digital technologies, throughout the system?

What do you see as the next

steps for the review?

It’s been running for 14 months and fol-lowing its interim report is now prepar-ing a final report. I believe the team has pulled together a very powerful set of ideas for mechanisms and new pathways and I am absolutely determined that we move quickly to implement the most rec-ommendations so that we can begin to make some quick advances.

I do not want to prejudice the independ-ence of that piece of work, led by Sir Hugh Taylor, but I think everybody can see that there is a real need to help accelerate those technologies that might help NHS Eng-land transform and modernise our health-care systems, for example, to avoid unnec-essary hospital admissions, so there are some obvious early wins in accelerating medical and diagnostic technologies.

Similarly, I think everyone can see there are real opportunities for NICE and NHS England to be given new flexibili-ties for innovative medicine assessment, for example, to take a new class of drugs where there is good genomic or clinical informatic data to fast-track them into a patient subset which we can guarantee or be very confident will deliver benefits. You can then develop new models of payment based on measuring the real out-come in those patients, in real time, with real disease, in real places. So, this review will be genuinely transformational.

NICE

How can NICE’s methodology better

take into account new medicines likely

to come to market in the coming years?

In particular those medicines that

are increasingly targeted at smaller

populations, such as precision or

personalised medicines?

NICE is one of the jewels in the crown of the UK drug discovery landscape and has led the world in the health economics of assessing the clinical and cost benefits of new drugs and other innovations. Central to its role is its independence, and I am absolutely determined not to undermine that in any way, but NICE was essentially created on the premise of a one-size-fits- all model of cost benefit.

It follows from the pace at which the new medicines landscape is changing that we need to give NICE the freedom to as-sess new drugs, particularly new cancer drugs, in a way that is much more precise. We also need to allow them and NHS England to build an assessment regime that is driven by real data, on real drugs, in real time, in real patients with real disease.

So, I hugely welcome the level of am-bition and engagement that Sir Andrew Dillon [the chief executive] and his team at NICE have shown in engaging with the AAR and am hopeful that we will see some recommendations to give NICE some new flexibilities.

Will the government give a mandate to

NICE to change its methodology to take

into account these new medicines?

It wouldn’t be appropriate for me to pre-scribe operational conditions to NICE, but we are clear that we want to accelerate patient access to the most beneficial, cost- effective and transformational medicines.

Do you think that NICE is appropriately

resourced to manage the scale of

medicines for approval and review?

As the minister for NICE, we have just completed its annual review and there was no complaint about under- resourcing. It is true, though, that the scale of progress in the drug-discovery field and the increas-ing variety of new drugs does test their existing systems, which is why I have in-vited the AAR and NICE to develop some new flexibilities.

CDF

Do you think that the proposal in

the NHS England consultation on

the Cancer Drugs Fund points to a

sustainable solution for funding of

cancer medicines?

An important question in this is: should we ring-fence particular commitments to innovative medicines? The Prime Minis-ter and I are very proud of the £1bn com-mitment to the CDF, not least because cancer has led the way in this quiet revo-lution of precision medicine.

We created the CDF to help with the challenges this is posing to the existing system. We also want to make sure that we use the CDF to accelerate UK and NHS leadership in 21st-century cancer therapy.

The NHS England review of the CDF has a vision of bringing it back down the stream from an end-of-process fund that largely funds drugs that have been re-jected by NICE at cost price – which has unsurprisingly attracted massive over-subscription – to a managed access fund.

This could fit into an accelerated access landscape more logically to accelerate the adoption of potentially transformational cancer therapies. This model is potentially very exciting.

How does the government plan to

work with NHS England and NICE to

manage and balance the priorities and

recommendations which are published

following the CDF consultation and

the AAR final report?

Given the clear alignment between the recent CDF consultation and the AAR, I have asked that NHS England and NICE work very closely with the AAR team so that we develop a joined-up logical land-scape that is very clear.

It is also important, in this global sec-tor, that innovators across the UK can see that there is a clear landscape for innova-tion, and that internationally the US re-forms that the FDA and the White House are pushing are complimentary to our re-forms here in the UK. It is my vision that the UK, after the AAR, should become the obvious gateway into the European market and indeed the first drugs in our early access to medicines scheme have indeed received faster European approval as a result. lThe conversation between Elisabeth Prchla and George Freeman MP took place in Westminster on 10 February 2016

“I’m confident the AAR review can be truly

transformational”


INTERVIEWt


VIEW FROM WESTMINSTER II

One of the most frustrating char-acteristics of the United King-dom is our inability to capital-ise on the huge strength of our science, innovation and quality

of research. How often have great ideas invented in the UK been developed in foreign countries that then make a much better fist of exploiting the potential?

Nowhere is this more apparent than in the NHS, with patients the real victims. We have an outstanding pharmaceutical industry in the UK. Billions of pounds are invested in research and development and many top medicines are developed. It’s the same for our diagnostic and health technology industry.

And yet, for all the undoubted strengths of the NHS, it has long been painfully slow to adopt new medicines and treat-ments. That was why the last Labour government developed NICE to speed up the introduction of clinically effective and cost-effective treatments and medi-cines. Backed by extra resources for the health service, NICE has become widely respected internationally.

But however good its methodology, it has never seemed to be able to respond to the challenges of cancer drugs, because of the timescale over which these are used by patients.

That is why the Cancer Drugs Fund (CDF) was established in 2011 to extend access to cancer drugs that were being turned down by NICE. Already, the CDF has helped more than 72,000 cancer pa-tients in England gain access to drugs not routinely funded.

Always intended as a short-term fix, the CDF closes to new drugs on 1 April. Because it became heavily oversubscribed, two processes of delisting of drugs previ-ously approved by the fund have already taken place. Thirty-two drugs covering 52 indications were removed, causing consternation among patient groups.

The government is consulting on pro-posals for a new cancer drugs fund, to launch in July, aimed at helping patients receive new treatments with genuine promise, while real-world evidence is collected for up to two years on how well they work in practice.

The proposal issued for public consul-tation outlines a system, fully integrated into the NICE appraisal process, where the CDF becomes a transitional fund – with clear criteria for entry and exit. The hope is that it will offer a new fast-track route to NHS funds, though much will depend on NICE being able to keep pace with the development of new medicines.

These proposals are matched by a more general review about access to medicines and treatments. Chaired by Sir Hugh Tay-lor, the former permanent secretary at the Department of Health, it emphasises the strength of our science base and the qual-ity of research. But Taylor warns that this could be undermined if research budg-ets are threatened and if the NHS cannot afford to support research, or if it fails to invest in change and innovation.

Its interim conclusions seek to give pa-tients a stronger voice and accelerate and manage entry into the NHS for the most significant emerging products. In addition

There is an alarming disparity between access to new medicines in the UK and the situation in other developed

countries, writes Lord Hunt

Time to end painfully slow NHS adoption

to expediting access to a select number of promising products, Sir Hugh wants the NHS to be an active partner in promot-ing innovation and giving practitioners an incentive to use new products.

But in the case of cancer drugs, two high hurdles have to be surmounted. The first is the NHS’s perverse approach to new medicines, which regards them as a bur-den. The second is to recognise that the fi-nancial plight facing the NHS is such that unless extra resources – possibly ring-fenced – can be found for new drugs, the proposals aren’t going to get anywhere.

Talking to senior managers in the NHS, you get the feeling that although they believe additional clinical staff and equipment are a good thing, any increase in demand on the drugs budget will be a disaster. Despite huge patient advances in the past fifty years, there is an alarm-ing disparity in access to new medicines in the UK compared to other developed countries. This cannot be divorced from finance. Recent OECD reports show that 24 countries spend more per share of GDP than the UK, and the top 18 spend more per capita. The next five years will bring a modest increase in NHS funds but a huge increase in demand, due to popu-lation increases and a rise in the number of vulnerable older patients.

Something has to give and all too often it will be the drugs budget. That is what makes the work on a new CDF and the Ac-celerated Access Review so important. lLord Hunt of Kings Heath is a Labour member of the House of Lords and shadow spokesperson on health


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